Product Description
Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 (CLN2), a paediatric neurodegenerative disease caused by a deficiency in TPP1. (Sourced from: https://pubmed.ncbi.nlm.nih.gov/28589525/)
Mechanisms of Action: TPP1 Modulator
Novel Mechanism: No
Modality: Coagulation Factor
Route of Administration: N/A
FDA Designation: *
Approval Status: Approved
Approved Countries: Australia | Austria | Belgium | Brazil | Canada | Croatia | Czech | Denmark | Estonia | European Medicines Agency | Finland | Germany | Greece | Hungary | Iceland | Ireland | Israel | Italy | Japan | Jordan | Korea | Latvia | Lithuania | Netherlands | Poland | Portugal | Romania | Slovakia | Slovenia | Sweden | Taiwan | Ukraine | United Kingdom | United States
Approved Indications: None
Known Adverse Events: None
Company: Biomarin
Company Location: NOVATO CA 94949
Company CEO: Jean-Jacques Bienaimé
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Recent & Upcoming Milestones
Highest Development Phases
Phase 2: Neuronal Ceroid-Lipofuscinosis|CLN2
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|
| NCT04476862 | N/A |
Active, not recruiting |
Neuronal Ceroid-Lipofuscinosis|CLN2 |
2030-08-31 |
2024-02-01 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments|Trial Status |
|
| NCT02963350 | N/A |
Approved for marketing |
CLN2 |
None |
2019-03-21 |
||
| CLN2 | P2 |
Active, not recruiting |
CLN2|Neuronal Ceroid-Lipofuscinosis |
2025-11-01 |
2023-09-14 |
Primary Endpoints|Treatments|Trial Status |
|
| NCT02678689 | P2 |
Completed |
CLN2|Neuronal Ceroid-Lipofuscinosis |
2022-04-20 |
15% |
2025-02-19 |
Primary Endpoints|Start Date|Treatments |
| NCT02485899 | P2 |
Completed |
Neuronal Ceroid-Lipofuscinosis|CLN2 |
2020-12-10 |
42% |
2022-08-25 |
Primary Endpoints|Treatments |
| 2014-003480-37 | P2 |
Unknown status |
Neuronal Ceroid-Lipofuscinosis|CLN2 |
2019-10-10 |
2022-03-13 |
Treatments |
|
| 2015-000891-85 | P2 |
Unknown status |
Neuronal Ceroid-Lipofuscinosis|CLN2 |
2019-07-05 |
2022-03-13 |
Treatments |
|
| NCT01907087 | P2 |
Completed |
Neuronal Ceroid-Lipofuscinosis|CLN2 |
2015-11-01 |
2019-03-19 |
Treatments |
|
| 2012-005430-11 | P2 |
Completed |
Neuronal Ceroid-Lipofuscinosis|CLN2 |
2014-11-30 |
2022-03-13 |
Treatments |